Life-Long AAV-Mediated CRISPR Genome Editing in Dystrophic Heart Improves Cardiomyopathy without Causing Serious Lesions in mdx Mice

Previous studies from others and us have demonstrated that CRISPR genome editing could offer a promising therapeutic strategy to restore dystrophin expression and function in the skeletal muscle and heart of Duchenne muscular dystrophy (DMD) mouse models. However, the long-term efficacy and safety o...

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Publicat a:Mol Ther
Autors principals: Xu, Li, Lau, Yeh Siang, Gao, Yandi, Li, Haiwen, Han, Renzhi
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2019
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6697345/
https://ncbi.nlm.nih.gov/pubmed/31129119
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.05.001
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